Recruiting
Early Phase 1

Ivacaftor

Sponsor:

University of Alabama at Birmingham

Code:

NCT03439865

Conditions

Chronic Rhinosinusitis (Diagnosis)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Ivacaftor

standard of care treatment

Study Details

Brief summary:

The purpose of this pilot study is to explore wither ivacaftor in refractory CRS patients will demonstrate safety and tolerability; restore CFTR-mediated Cl- secretions as measured by EDSPD testing; produce detectable improvements in validated measures of CRS including the SNOT-22 questionnaire, Lund-MacKay CT scan grading, and Lund-Kennedy endoscopic scores; and provide beneficial effects on readily measured markers of sinonasal inflammation and infection (IP-10, IL-8, and Pseudomonas CFUs).

Conditions

Chronic Rhinosinusitis (Diagnosis)

Study ID

NCT03439865

Start date

May 2, 2019

Status verified date

Apr, 2026

Completion date

May 1, 2027

Anticipated

Primary completion date

Apr, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 18 years of age
  • Patient has provided informed consent
  • Diagnosis of CRS made by one of the investigators
  • Standard of care CT scan with definitive demonstration of isolated or diffuse mucosal thickening, bone changes, and air fluid levels, obtained within 30 days of treatment
  • Positive culture of at least one gram negative bacteria (e.g. Pseudomonas, E. coli, Steenotrophomonas) within 30 days prior to testing
  • Previous surgery with (at least) exposed maxillary and ethmoid sinuses
  • Ability to perform EDSPD testing such that nasal cavity space and sinus openings are sufficient for catheter placement
  • Negative pregnancy test for females of childbearing potential within 72 hours of testing and start of study treatment

Exclusion Criteria:

  • < 18 years of age
  • Acute illness other than sinusitis within 2 weeks before start of study treatment that, in the opinion of the investigator, would preclude participation
  • Currently taking medications that are moderate or strong CP3A inhibitors
  • History of asthma attack requiring emergency room visit or treatment with oral steroids within 2 months prior to study treatment
  • History of solid organ or hematological transplantation
  • History of known immunodeficiency, autoimmune or granulomatous disorder
  • Serum creatinine > 1.5x upper normal limit
  • Abnormal liver function, as defined by serum AST > 2x upper normal limit, serum ALT > 2x upper normal limit, Alkaline phosphatase > 2x upper normal limit, Total bilirubin > 2x upper normal limit
  • Women who are pregnant or breastfeeding

Study Design

Enrollment

20 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: standard of care treatment + ivacaftor

topical nasal steroid spray and culture-directed antibiotics + ivacaftor 150 mg tablet

placebo comparator: standard of care treatment

topical nasal steroid spray and culture-directed antibiotics

Interventions

Ivacaftor

150 mg tablet PO BID x 14 days

standard of care treatment

topical nasal steroid spray and culture-directed antibiotics x 14 days

Primary outcome measure

  • Improvement in quality of life measures [ Time Frame: Screening to Day 30 ]

Central Contacts and Locations

Central contacts

Norma Miller, RN

(205) 975-6169ncmiller@uabmc.edu

Locations

University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Norma Miller, RN

(205) 975-6169ncmiller@uabmc.edu

More Information

Sponsor

University of Alabama at Birmingham

Last update posted

Apr 22, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Alabama at Birmingham on 2026-04-22.